CN107922942B - 三组分crispr/cas复合系统及其用途 - Google Patents

三组分crispr/cas复合系统及其用途 Download PDF

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CN107922942B
CN107922942B CN201680027107.2A CN201680027107A CN107922942B CN 107922942 B CN107922942 B CN 107922942B CN 201680027107 A CN201680027107 A CN 201680027107A CN 107922942 B CN107922942 B CN 107922942B
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sequence
polynucleotide
puf
protein
dna
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CN107922942A (zh
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王皓毅
A·程
N·吉勒特
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Jackson Laboratory
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    • C—CHEMISTRY; METALLURGY
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
CN201680027107.2A 2015-03-13 2016-03-09 三组分crispr/cas复合系统及其用途 Active CN107922942B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562132644P 2015-03-13 2015-03-13
US62/132,644 2015-03-13
US201562221249P 2015-09-21 2015-09-21
US62/221,249 2015-09-21
PCT/US2016/021491 WO2016148994A1 (fr) 2015-03-13 2016-03-09 Système de complexe cas/crispr à trois constituants et utilisations de ce dernier

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CN107922942A CN107922942A (zh) 2018-04-17
CN107922942B true CN107922942B (zh) 2021-08-31

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US (2) US11434484B2 (fr)
EP (2) EP3858992A1 (fr)
JP (3) JP6588995B2 (fr)
KR (1) KR102004076B1 (fr)
CN (1) CN107922942B (fr)
AU (2) AU2016233648B2 (fr)
CA (2) CA3091771A1 (fr)
ES (1) ES2886901T3 (fr)
HK (2) HK1249545A1 (fr)
IL (2) IL254428B (fr)
SG (2) SG10202008443UA (fr)
WO (1) WO2016148994A1 (fr)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 プレジデント アンド フェローズ オブ ハーバード カレッジ ヌクレアーゼ切断特異性の評価および改善
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
WO2016022363A2 (fr) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Protéines cas9 comprenant des intéines dépendant de ligands
US20190225955A1 (en) 2015-10-23 2019-07-25 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11780895B2 (en) 2016-09-13 2023-10-10 The Jackson Laboratory Targeted DNA demethylation and methylation
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592777A1 (fr) 2017-03-10 2020-01-15 President and Fellows of Harvard College Éditeur de base cytosine à guanine
US11268082B2 (en) 2017-03-23 2022-03-08 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
WO2019014564A1 (fr) 2017-07-14 2019-01-17 Editas Medicine, Inc. Systèmes et procédés d'intégration ciblée et d'édition du génome et détection de celle-ci à l'aide de sites d'amorçage intégrés
WO2019023680A1 (fr) 2017-07-28 2019-01-31 President And Fellows Of Harvard College Procédés et compositions pour l'évolution d'éditeurs de bases à l'aide d'une évolution continue assistée par phage (pace)
WO2019139645A2 (fr) 2017-08-30 2019-07-18 President And Fellows Of Harvard College Éditeurs de bases à haut rendement comprenant une gam
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
KR102013798B1 (ko) * 2017-10-25 2019-08-23 성균관대학교산학협력단 노화 모델 제조 방법 및 이에 의해 제조된 세포 또는 동물 노화 모델
EP3755726A4 (fr) * 2018-02-23 2022-07-20 Shanghaitech University Protéines de fusion pour édition de base
WO2019183630A2 (fr) * 2018-03-23 2019-09-26 The Trustees Of Columbia University In The City Of New York Édition de gènes pour maladies autosomiques dominantes
EP3575401A1 (fr) * 2018-05-29 2019-12-04 Ecole Polytechnique Federale De Lausanne (Epfl) Méthodes de traitement ou de prévention d'une maladie liée au vieillissement
CN109055375B (zh) * 2018-07-26 2021-11-12 东南大学 一种crispr辅助反式增强子激活基因表达的方法及其应用
WO2020041172A1 (fr) * 2018-08-21 2020-02-27 The Jackson Laboratory Procédés et compositions de recrutement de protéines de réparation d'adn
CA3130488A1 (fr) 2019-03-19 2020-09-24 David R. Liu Procedes et compositions pour l'edition de sequences nucleotidiques
CN114555826A (zh) * 2019-08-16 2022-05-27 杰克逊实验室 非重复基因组基因座的活细胞成像
WO2021138560A2 (fr) 2020-01-02 2021-07-08 The Trustees Of Columbia University In The City Of New York Activation transcriptionnelle de crispr-cas programmable et portable dans des bactéries
CN115485305A (zh) * 2020-02-28 2022-12-16 杰克逊实验室 癌细胞中裂解基因的激活
GB2614813A (en) 2020-05-08 2023-07-19 Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
EP4176058A1 (fr) 2020-08-07 2023-05-10 The Jackson Laboratory Compositions et procédés d'insertion de séquence ciblée
WO2022272294A1 (fr) * 2021-06-23 2022-12-29 The Board Of Trustees Of The Leland Stanford Junior University Compositions et procédés pour un recrutement efficace des retrons au niveau des cassures d'adn
CN113637658A (zh) * 2021-08-19 2021-11-12 中国农业科学院棉花研究所 基于dCas9-oToV的基因转录系统及其应用
CN114058641B (zh) * 2022-01-17 2022-04-01 苏州大学 一种载体系统和应用及通过其降解目的蛋白的方法
US20230340582A1 (en) * 2022-04-07 2023-10-26 Trustees Of Boston University Compositions and methods relating to nucleic acid interaction reporters
WO2023224327A1 (fr) * 2022-05-20 2023-11-23 한국생명공학연구원 Système et procédé pour augmenter l'efficacité d'édition génique de microalgues à l'aide d'autolysine

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011160052A2 (fr) * 2010-06-18 2011-12-22 The University Of North Carolina At Chapel Hill Procédés et compositions associés à des arn-endonucléases spécifiques d'une séquence

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585362A (en) 1989-08-22 1996-12-17 The Regents Of The University Of Michigan Adenovirus vectors for gene therapy
DE69233013T2 (de) 1991-08-20 2004-03-04 The Government Of The United States Of America As Represented By The Secretary Of National Institute Of Health, Office Of Technology Transfer Adenovirus vermittelter gentransfer in den gastrointestinaltrakt
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
FR2688514A1 (fr) 1992-03-16 1993-09-17 Centre Nat Rech Scient Adenovirus recombinants defectifs exprimant des cytokines et medicaments antitumoraux les contenant.
EP1024198A3 (fr) 1992-12-03 2002-05-29 Genzyme Corporation Vecteurs pseudo-adenoviraux pour la thérapie génique des hémophilies
DE69434486T2 (de) 1993-06-24 2006-07-06 Advec Inc. Adenovirus vektoren für gentherapie
ES2256842T4 (es) 1993-10-25 2007-02-01 Canji, Inc. Vector de adenovirus recombinante y metodos de uso.
US7078387B1 (en) 1998-12-28 2006-07-18 Arch Development Corp. Efficient and stable in vivo gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors
EP1083231A1 (fr) 1999-09-09 2001-03-14 Introgene B.V. Promoteur spécifique des cellules musculaires lisses, et applications
US7169874B2 (en) 2001-11-02 2007-01-30 Bausch & Lomb Incorporated High refractive index polymeric siloxysilane compositions
EP2393484A1 (fr) 2009-02-06 2011-12-14 Egalet Ltd. Composition à libération immédiate résistant à une maltraitance par prise d'alcool
US9580714B2 (en) * 2010-11-24 2017-02-28 The University Of Western Australia Peptides for the specific binding of RNA targets
PE20190844A1 (es) 2012-05-25 2019-06-17 Emmanuelle Charpentier Modulacion de transcripcion con arn de direccion a adn generico
BR112015000150A2 (pt) 2012-07-06 2017-06-27 Egalet Ltd composições farmacêuticas dissuasoras de abuso de liberação controlada
US11780895B2 (en) * 2016-09-13 2023-10-10 The Jackson Laboratory Targeted DNA demethylation and methylation

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011160052A2 (fr) * 2010-06-18 2011-12-22 The University Of North Carolina At Chapel Hill Procédés et compositions associés à des arn-endonucléases spécifiques d'une séquence

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
《CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering》;Mali Prashant等;《NATURE BIOTECHNOLOGY》;20130930;第31卷(第9期);第833-838页 *
《Casilio: a versatile CRISPR-Cas9-Pumilio hybrid for gene regulation and genomic labeling》;Cheng Albert W等;《CELL RESEARCH》;20160115;第26卷(第2期);第254-257页 *
《Engineering Complex Synthetic Transcriptional Programs with CRISPR RNA Scaffolds》;Zalatan Jesse G等;《CELL》;20150115;第160卷(第1-2期);第339-350页 *

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JP6588995B2 (ja) 2019-10-09
IL276894A (en) 2020-10-29
KR102004076B1 (ko) 2019-07-25
CA2979567A1 (fr) 2016-09-22
KR20170124601A (ko) 2017-11-10
IL276894B (en) 2022-12-01
US11434484B2 (en) 2022-09-06
WO2016148994A1 (fr) 2016-09-22
EP3268472A1 (fr) 2018-01-17
US20180094257A1 (en) 2018-04-05
IL276894B2 (en) 2023-04-01
EP3268472B1 (fr) 2021-05-05
AU2016233648B2 (en) 2019-11-07
CA2979567C (fr) 2020-10-13
AU2020200256B2 (en) 2022-03-31
HK1249545A1 (zh) 2018-11-02
HK1254126A1 (zh) 2019-07-12
AU2016233648A1 (en) 2017-10-05
JP2019176877A (ja) 2019-10-17
CA3091771A1 (fr) 2016-09-22
IL254428A0 (en) 2017-11-30
JP2021036899A (ja) 2021-03-11
IL254428B (en) 2020-08-31
CN107922942A (zh) 2018-04-17
SG10202008443UA (en) 2020-10-29
WO2016148994A8 (fr) 2016-11-03
ES2886901T3 (es) 2021-12-21
EP3858992A1 (fr) 2021-08-04
SG11201707393SA (en) 2017-10-30
US20230071712A1 (en) 2023-03-09
JP2018507713A (ja) 2018-03-22
AU2020200256A1 (en) 2020-02-06
JP6867440B2 (ja) 2021-04-28

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